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Extension Study of PRX-102 for up to 60 Months

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01981720
Recruitment Status : Enrolling by invitation
First Posted : November 11, 2013
Last Update Posted : February 5, 2018
Information provided by (Responsible Party):

Brief Summary:
To evaluate the ongoing safety, tolerability, and efficacy parameters of PRX-102 in adult Fabry patients who have successfully completed treatment with PRX-102 in studies PB-102-F01 and PB-102-F02. Patients will be enrolled to receive PRX-102 at the dose of, 1.0 mg/kg, as an intravenous infusion every 2 weeks (+/- 3 days). The duration of treatment will be up to 60 months or until PRX-102 is commercially available to the subject at the discretion of the Sponsor and no less than 36 months.

Condition or disease Intervention/treatment Phase
Fabry Disease Biological: PRX-102 (pegunigalsidase alfa) Phase 1 Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 16 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multi Center Extension Study of PRX-102 Administered by Intravenous Infusions Every 2 Weeks for up to 60 Months to Adult Fabry Patients
Actual Study Start Date : December 2013
Estimated Primary Completion Date : March 2021
Estimated Study Completion Date : March 2021

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arm Intervention/treatment
Experimental: 1.0 mg/kg
PRX-102 (pegunigalsidase alfa) 1.0 mg/kg IV every 2 weeks (+/- 3 days)
Biological: PRX-102 (pegunigalsidase alfa)
PRX-102 1 mg/kg every 2 weeks
Other Names:
  • pegunigalsidase alfa
  • Recombinant human alpha galactosidase-A

Primary Outcome Measures :
  1. Number of participants with treatment-related adverse events as assessed by CTCAE v4.03 [ Time Frame: Every two weeks for 60 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Completion of study PB-102-F02
  • The patient signs informed consent
  • Female patients of child-bearing potential agree to use a medically acceptable method of contraception, not including the rhythm method. Acceptable methods of contraception include hormonal products, intrauterine device, or male or female condoms. Contraception should be used throughout the duration of the study and for 3 months after termination of treatment.

Exclusion Criteria:

  • Pregnant or nursing
  • Presence of any medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator and/or Medical Director, would interfere with the patient's compliance with the requirements of the study

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01981720

United States, Florida
University of Florida
Gainesville, Florida, United States, 32611
United States, Iowa
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States, 52242
United States, Texas
Institute of Metabolic Disease
Dallas, Texas, United States, 75226
United States, Virginia
O & O Alpan
Fairfax, Virginia, United States, 22030
Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C)
Asunción, Paraguay
Hospital de Dia Quiron Zaragoza
Zaragoza, Spain, 50012
United Kingdom
The Royal Free Hospital
London, United Kingdom, NW3 2QG
Sponsors and Collaborators
Study Director: Raul Chertkoff, MD Protalix Inc.

Responsible Party: Protalix
ClinicalTrials.gov Identifier: NCT01981720     History of Changes
Other Study ID Numbers: PB-102-F03
First Posted: November 11, 2013    Key Record Dates
Last Update Posted: February 5, 2018
Last Verified: February 2018

Studies a U.S. FDA-regulated Drug Product: Yes

Keywords provided by Protalix:
pegunigalsidase alfa
Fabry Disease

Additional relevant MeSH terms:
Fabry Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders